S36ɳÁú»á

¿ËÈÕ£¬£¬£¬
£¬£¬£¬ÖÐɽ´óѧÖÐɽÑÛ¿ÆÖÐÐĽÌÊÚÕÅÐãÀ¼ÍŶÓÓë½£ÇÅ´óѧ½ÌÊÚPatrick Yu-Wai ManÏàÖú£¬£¬£¬
£¬£¬£¬Ê¹ÓÃCRISPR-Cas9»ùÒò±à¼­ÊÖÒÕ°ÐÏòÇóý̼Ëáôûø2£¨Car2£©»ùÒò£¬£¬£¬
£¬£¬£¬ÔÚСÊóÄ£×ÓÉϵ¥´Î¸øÒ©»ñµÃÒ»Á¬½µµÍÑÛѹÁ½¸öÔÂÒÔÉϵÄÏÔÖø×÷ÓÃ£¬£¬£¬
£¬£¬£¬ÎªÇà¹âÑÛÌṩÁ˹ãÆס¢³¤Ð§½µµÍÑÛѹ»ùÒòÖÎÁƵĿÉÄÜÐÔ¡£¡£¡£¡£Ïà¹ØЧ¹û½ÒÏþÓÚ¡¶Ï¸°û±¨µÀҽѧ¡·¡£¡£¡£¡£

¡°ÎÒÃÇÍ»ÆÆÁ˹Űå»ùÒòÖÎÁÆÑо¿¿ò¼Ü£¬£¬£¬
£¬£¬£¬´ÓÁÙ´²ÏÖʵÐèÇó³ö·¢£¬£¬£¬
£¬£¬£¬Ñ°ÕÒµ½ÊʺÏËùÓÐÇà¹âÑÛÀàÐ͵ÄCar2»ùÒò¾ÙÐйãÆ×»ùÒòÖÎÁÆÑо¿¡£¡£¡£¡£¡±ÂÛÎÄÅäºÏͨѶ×÷ÕßÕÅÐãÀ¼ÌåÏÖ£¬£¬£¬
£¬£¬£¬¸ÃÑо¿Ð§¹û¾ßÓм«´óµÄÁÙ´²×ª»¯Ç±Á¦£¬£¬£¬
£¬£¬£¬»ùÒò±à¼­Ò©ÎïÓÐÍûÌæ»»¹Å°åÒ©ÎïÖÎÁÆÊÖ¶Î£¬£¬£¬
£¬£¬£¬ÊµÏÖ¡°One shot gene therapy¡ª¡ªµ¥´Î¸øÒ©£¬£¬£¬
£¬£¬£¬³¤Ð§½µÑ¹¡±µÄÄ¿µÄ£¬£¬£¬
£¬£¬£¬ÎªËùÓÐÀàÐ͵ÄÇà¹âÑÛ»¼Õß¼õÇá¼²²¡¼ç¸º¡£¡£¡£¡£

Çà¹âÑÛÊÇÈ«Çò¹æÄ£ÄÚµÚÒ»´ó²»¿ÉÄæÖÂäÐÔÑÛ²¡¡£¡£¡£¡£»£»£»
£»£»ùÒò±à¼­Äܹ»Ìṩµ¥´Î¸ÉÔ¤¡¢³¤Ð§ÖÎÁƵĿÉÄÜ£¬£¬£¬
£¬£¬£¬ÊÇÇà¹âÑÛÀíÏëµÄÖÎÁƹ¤¾ß¡£¡£¡£¡£ÕÅÐãÀ¼ÍŶÓÀú¾­3Äê¶àʱ¼äµÄDZÐÄÑо¿£¬£¬£¬
£¬£¬£¬´ÓÁÙ´²½µÑÛѹҩÎïµÄ×÷ÓûúÖƳö·¢£¬£¬£¬
£¬£¬£¬Ñ°ÕÒµ½ÊʺÏËùÓÐÇà¹âÑÛÀàÐ͵ÄCar2»ùÒò¾ÙÐйãÆ×»ùÒòÖÎÁÆÑо¿£¬£¬£¬
£¬£¬£¬Ê¹ÓÃCRISPR-Cas9ÊÖÒÕÇóýCar2»ùÒò£¬£¬£¬
£¬£¬£¬Í¨¹ýµ¥´Î²£Á§ÌåÇ»×¢Éä£¬£¬£¬
£¬£¬£¬ÔÚСÊóÄ£×ÓÖлñµÃ³¤´ïÁ½¸öÔµĽµÑÛѹЧ¹û£¬£¬£¬
£¬£¬£¬ÎªÇà¹âÑÛÌṩÁ˹ãÆ×»ùÒòÁÆ·¨µÄ¿ÉÄÜÐÔ¡£¡£¡£¡£

ÕÅÐãÀ¼ÍŶÓÊ×ÏÈÕë¶ÔСÊóCar2»ùÒòÉè¼ÆÁËÁùÌõsgRNAÐòÁÐ£¬£¬£¬
£¬£¬£¬²¢ÔÚÌåÍâʹÓÃT7E1ʵÑéÁ¬ÏµÉî¶È²âÐòÊÖÒÕÆÀ¹À¸÷sgRNAµÄ»ùÒò±à¼­Ð§ÂÊ¡£¡£¡£¡£ÎªÁ˽øÒ»²½Ìá¸ß»ùÒò±à¼­Ð§ÂÊ£¬£¬£¬
£¬£¬£¬ÍŶÓɸѡ³öÁ½ÌõsgRNA²¢¹¹½¨Ë«°ÐµãCRISPRϵͳ£¬£¬£¬
£¬£¬£¬Ð§¹ûÅú×¢»ùÒò±à¼­Ð§Âʱ»ÏÔÖøÌáÉýÖÁ97.3%¡£¡£¡£¡£½Ó×Å£¬£¬£¬
£¬£¬£¬ÎªÁËÑéÖ¤Car2»ùÒòÇóýÊÇ·ñÄܹ»½µµÍÑÛѹ£¬£¬£¬
£¬£¬£¬¸ÃÍŶӽ«Ë«°ÐµãCRISPRϵͳ°ü×°ÖÁShH10ѪÇåÐ͵ÄÏÙÏà¹Ø²¡¶¾£¨AAV£©ÔØÌåÖС£¡£¡£¡£ShH10-AAV¶ÔÈÏÕæ·¿Ë®ÌìÉúµÄ½Þ×´Ìå×éÖ¯ÓкܸߵÄÇ׺ÍÐÔ¡£¡£¡£¡£Í¨¹ýµ¥´Î²£Á§ÌåÇ»×¢Éä£¬£¬£¬
£¬£¬£¬ÍŶÓ֤ʵ˫°ÐµãCRISPRϵͳÄܹ»°ÐÏò½Þ×´Ìå×éÖ¯£¬£¬£¬
£¬£¬£¬ïÔÌ­CAR2ÂѰ׵ıí´ï¡£¡£¡£¡£ÑÛѹ¼à²âЧ¹ûÒ²Åú×¢Car2»ùÒòÇóýÄܹ»ÏÔÖø½µµÍÕý³£Ð¡ÊóµÄ»ùÏßÑÛѹ£¬£¬£¬
£¬£¬£¬Ïà½ÏÓÚ±ÈÕÕ×éïÔÌ­Ô¼3mmHg£¬£¬£¬
£¬£¬£¬²¢ÇÒÒ»Á¬Á½¸öÔÂÒÔÉÏ¡£¡£¡£¡£

ΪÁ˽øÒ»²½Ì½Ë÷Car2»ùÒòÇóý¶ÔÇà¹âÑÛµÄÖÎÁÆЧ¹û£¬£¬£¬
£¬£¬£¬Ñо¿ÍŶӽÓÄÉÁËDBA/2JСÊóºÍ´ÅÖéÓÕµ¼ÂýÐÔ¸ßÑÛѹģ×ÓÁ½ÖÖʵÑéÐÔÇà¹âÑÛÄ£×Ó¡£¡£¡£¡£Ð§¹ûÅú×¢Car2»ùÒòÇóýÄܹ»ÏÔÖø½µµÍÇà¹âÑÛÄ£×ÓСÊóµÄÑÛѹ£¬£¬£¬
£¬£¬£¬²¢ÇÒÄܹ»ÑÓ»ºÉõÖÁ×èÖ¹¸ßÑÛѹ´øÀ´µÄ×é֯ѧËðÉË¡£¡£¡£¡£±ðµÄ£¬£¬£¬
£¬£¬£¬ÔÚÂýÐÔ¸ßÑÛѹģ×ÓÖÐ£¬£¬£¬
£¬£¬£¬ÒÔÁÙ´²³£ÓýµÑÛѹҩÎïÅÉÁ¢Ã÷£¨²¼ÁÖ×ô°·£©µÎÑÛÒº×÷±ÈÕÕ£¬£¬£¬
£¬£¬£¬·¢Ã÷Car2»ùÒòÇóý½µÑÛѹЧ¹ûÒªÂÔÓÅÓÚÅÉÁ¢Ã÷£¬£¬£¬
£¬£¬£¬¸Ã·¢Ã÷¼«´óÔö½øÁË»ùÒò±à¼­Ò©ÎïÌæ»»¹Å°åÒ©ÎïÖÎÁÆÇà¹âÑ۵ĿÉÄÜÐÔ¡£¡£¡£¡£

Ö»¹ÜÑÛÇòÒÀ¸½¡°ÃâÒß¿íÃ⡱ÊÇÀíÏëµÄ»ùÒòÖÎÁÆ°ÐÆ÷¹Ù£¬£¬£¬
£¬£¬£¬µ«ÓÉÓÚAAV²¡¶¾È±·¦×éÖ¯ÌØÒìÐÔ£¬£¬£¬
£¬£¬£¬ÎÞ·¨×èÖ¹ÊÓÍøĤµÄתȾ£¬£¬£¬
£¬£¬£¬Òò´ËÕÅÐãÀ¼ÍŶÓͨ¹ý×é֯ѧºÍ¹¦Ð§Ñ§ÆÀ¹ÀCar2»ùÒòÇóýµÄÇå¾²ÐÔºÍDZÔÚ¸±×÷Óᣡ£¡£¡£OCTÒÔ¼°ERGЧ¹ûÅú×¢£¬£¬£¬
£¬£¬£¬Car2»ùÒòÇóýδÒýÆðDZÔڵIJ»Á¼·´Ó¦¡£¡£¡£¡£